In a groundbreaking medical first, researchers in the Netherlands have successfully edited mitochondrial DNA something long believed to be beyond the reach of modern gene-editing tools like CRISPR. Published in PLOS Biology, the study marks a historic milestone for genetic medicine, offering the first real glimmer of hope for patients suffering from rare, inherited mitochondrial diseases that were previously considered untreatable.
The secret weapon? A revolutionary tool called DdCBE (double-stranded DNA deaminase-derived cytosine base editor). Unlike CRISPR, which can’t penetrate mitochondria, DdCBE doesn’t require cutting the DNA strand. Instead, it rewrites specific faulty genetic letters with precision, reducing the risk of dangerous off-target mutations. When tested on liver and skin cells from real patients, the edited cells regained mitochondrial function and the changes remained stable over time.
To top it off, scientists used lipid nanoparticles and mRNA the same delivery method used in COVID-19 vaccines to insert the gene-editing tool, making the therapy not only powerful but also highly targeted and minimally invasive. While human trials are still a few years away, this breakthrough paves the way for future treatments that were once only dreamed of. For thousands of families facing life-limiting mitochondrial disorders, this is no longer just research it’s the beginning of real, tangible hope.
Source: Indi P. Joore et al., PLOS Biology (2025)
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